#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…

Adding Azilect (rasagiline) — a medication already approved to treat Parkinson’s disease — to Rilutek (riluzole) may be able to slow the advancement of fast-progressing amyotrophic lateral sclerosis (ALS), a Phase 2 trial suggests. However, its positive safety results are being challenged by other physicians, who raise concerns that…

Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are caused by different patterns of inflammation in the brain, which may help explain why these two illnesses are so distinct. The German study, “Different neuroinflammatory profile in amyotrophic lateral sclerosis and frontotemporal dementia is linked to the…

My first symptomatic expression of ALS may have been as I left a football game. I fell flat on my face. At the time, I blamed it on the sun and the beer, although that combination in the same dosages had not felled me before. Shortly after, I became prone…

A newly developed method can help characterize the types of misfolded proteins present in patients with amyotrophic lateral sclerosis (ALS), according to findings in a thesis. The recently published doctoral thesis, titled “Structural investigation of SOD1 aggregates in ALS: identification of prion strains using anti-peptide antibodies,” was written…

Sometimes living with ALS makes me feel like I’m competing in the Olympics. I wake up in the morning and wonder how my body will perform that day. Then I lie awake at night rehashing all the things that went wrong. The voices in my head often sound…

Retrotope is providing its investigational fatty acid being developed for Friedreich’s ataxia, RT001, to people with amyotrophic lateral sclerosis (ALS) under an expanded access program, also called “compassionate use.” Individual patients with life-threatening or severely debilitating diseases can petition the U.S. Food and Drug Administration, through their physicians,…

Human spinal cord neural stem cells (NSC), created using an innovative method, were seen to regenerate functional neurons in the damaged tissue of rats with spinal injuries, according to researchers. Their study, “Generation and post-injury integration of human spinal cord neural stem cells,” was published in the journal…

Amyotrophic lateral sclerosis’ toll on the thinking abilities and behavior of patients is as progressive and damaging as the disease’s physical toll, researchers in the U.K. report, with  ALS-specific cognitive and behavioral deficits showing an impact even in early stages and worsening as the disease advances. ALS is characterized by the…

Depleting a protein called Staufen1 improved motor function and lowered protein aggregation in a mouse model of spinocerebellar ataxia type 2 (SCA2). The findings suggest that targeting Staufen1 could lead to therapies for amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. The study “Staufen1 links…